New hemangioma treatments can have positive effects on infants

August 9, 2014

‘Hemangiomas can cause stress and concern for parents, but luckily, most of these lesions do not require intervention, so we can reassure the parents,’ Esteban Fernandez Faith, MD, said. ‘Although they are benign, these are important because some could lead to complications.’

Vascular anomalies can have a traumatic effect as they may cause medical complications and mar a person’s appearance, but this is particularly true of their subset — hemangiomas — that appear in the weeks after a baby is born. New advances in their treatment are improving outcomes, but timing of treatment is vital.

“One of most important issues is education for pediatricians and primary care physicians to let them know there are more and better treatment options. These used to be lesions for which therapies were limited and we would just have to deal with the complications, but now we have better and safer treatment options so we can be more proactive,” said Esteban Fernandez Faith, MD, a pediatric dermatologist. “Education about the critical time frame when we would like to see these patients will help prevent complications. That is an important part of our job.”

Dr. Fernandez Faith discussed the advances Friday during his Plenary presentation, “Update on Hemangiomas and Vascular Anomalies.” He is an assistant professor at The Ohio State University and on staff at Nationwide Children’s Hospital, Columbus, Ohio.

Hemangiomas are benign neoplasms that usually are not present or fully developed at birth but start to appear in the first few weeks of life. They go through a rapid proliferative stage in the first three months of life, but can grow for six months.

“Hemangiomas can cause stress and concern for parents, but luckily, most of these lesions do not require intervention, so we can reassure the parents,” he said. “Although they are benign, these are important because some could lead to complications.

“The potential complications include ulceration, bleeding, and ocular problems when hemangiomas occur around the eyes. There also can be airway hemangiomas. If they appear around the mouth or the lips they can cause problems with feeding,” Dr. Fernandez Faith said. “Hemangiomas on the face can cause an anatomic deformity, and these are the patients with whom we are concerned in regard to their psychosocial development.”

“Potentially there are not only medical or physical complications, but because they tend to be very visible, they can affect the patient’s quality of life and psychosocial development. That is a critical part of a child’s development.”

An important milestone in our understanding of vascular anomalies began with a classification system developed in the 1980s. Knowledge of these lesions has progressed and evolved since then, with the most recent update of the classification proposed earlier this year by the International Society for the Study of Vascular Anomalies.

“Another milestone was the discovery of an immunochemical marker GLUT1 for hemangiomas, which sets them apart from a histochemical standpoint,” he said. “This is now being used in clinical practice to differentiate hemangiomas from other vascular lesions. There have been several studies looking at where these lesions come from, their origin, and the molecular mechanisms involved.”

These advances have led to the development of a mouse model to better understand the pathogenesis of hemangiomas. “It is also a good way to test different treatment options,” Dr. Fernandez Faith said.

Propranolol, a beta blocker, has been used to treat hemangiomas since 2008. In March, a pediatric formulation of propranolol designed for infants, Hemangeol (a propranolol hydrochloride oral solution), was approved by the Food and Drug Administration. Hemangeol is given two times a day, usually for several months, to get the most benefit, he said.

“Propranolol was a game-changer for the way we manage and treat these patients,” Dr. Fernandez Faith said. “One of the advantages of propranolol is that it stops the growth of the hemangioma, but we also know that propranolol makes them start to involute much quicker so there are better results at the end.”

A topical beta blocker, timolol, also is being used to treat hemangiomas. Although the medication was developed to treat glaucoma, it is being used off-label to treat smaller, superficial lesions.

Vascular malformations are another category of vascular anomalies, which in general have a slower progression than hemangiomas. On the extremities, vascular malformations can cause mobility limitations, pain, swelling, and inflammation. Malformations on the neck or trunk can affect internal organs, such as the upper airway and lungs.

“One of the most important advances in the area of vascular anomalies is the use of rapamycin for patients who have a complicated vascular anomaly,” Dr. Fernandez Faith said. “A recent study shows that this proved to be beneficial for some of these patients. Rapamycin is used mainly for transplant rejection, and it is a medication that could have important complications or side effects. Rapamycin is gaining significant attention in this field. There is still not an indication for vascular anomalies, so there are some concerns about side effects, particularly in young children.”

An animal model has been developed for venous malformations, which will help researchers better understand their molecular mechanisms and potentially develop more treatment options, he said.

“Another area of research with vascular anomalies is to try to determine biomarkers or ways to screen these patients and determine which are more likely to develop complications in the future so we can treat them before the complications develop,” Dr. Fernandez Faith said. “This is an exciting time in the study of vascular anomalies. Because of the pace of research and the advances in this area, the way we approach and manage these patients has changed quite significantly and rapidly.”

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